The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: A 17.5 year follow-up.
The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: A 17.5 year follow-up.
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DOI：
10.1002/ajh.21699
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发表时间：
2010-06
影响因子：
12.8
通讯作者：
Waclawiw, Myron A.
中科院分区：
文献类型：
--
作者：
Steinberg, Martin H.;McCarthy, William F.;Castro, Oswaldo;Ballas, Samir K.;Armstrong, F. Danny;Smith, Wally;Ataga, Kenneth;Swerdlow, Paul;Kutlar, Abdullah;DeCastro, Laura;Waclawiw, Myron A.
一项随机对照临床试验确定了短期使用羟基脲治疗成人镰状细胞性贫血的有效性和安全性。为了检查长期使用羟基脲的风险和益处，本试验对患者进行了17.5年的随访，在此期间他们可以开始或停止使用羟基脲。本次随访的目的是寻找不良后果和估计死亡率。对于每个结局和死亡率，计算精确的95%置信区间，或在α = 0.05水平上进行检验（p值<0.05为有统计学意义）。虽然整个研究队列的死亡率很高（43.1%，每100人年4.4人），但长期暴露于羟基脲的个体死亡率降低。生存曲线显示，长期接触的死亡率显著降低。24%的死亡是由于肺部并发症；87.1%的患者从未服用羟脲或服用时间<5年。卒中、脏器功能障碍、感染及恶性肿瘤发生率各组相似。我们的研究结果表明，长期使用羟基脲是安全的，并且可能降低死亡率，但由于伦理上的考虑，我们的研究结果不再是随机研究的结果。
A randomized, controlled clinical trial established the efficacy and safety of short-term use of hydroxyurea in adult sickle cell anemia. To examine the risks and benefits of long-term hydroxyurea usage, patients in this trial were followed for 17.5 years during which they could start or stop hydroxyurea. The purpose of this follow-up was to search for adverse outcomes and estimate mortality. For each outcome and for mortality, exact 95% confidence intervals were calculated, or tests were conducted at α = 0.05 level (p-value <0.05 for statistical significance). Although the death rate in the overall study cohort was high (43.1%; 4.4 per 100 person-years), mortality was reduced in individuals with long-term exposure to hydroxyurea. Survival curves demonstrated a significant reduction in deaths with long-term exposure. Twenty-four percent of deaths were due to pulmonary complications; 87.1% occurred in patients who never took hydroxyurea or took it for <5 years. Stroke, organ dysfunction, infection and malignancy were similar in all groups. Our results, while no longer the product of a randomized study because of the ethical concerns of withholding an efficacious treatment, suggest that long-term use of hydroxyurea is safe and might decrease mortality.
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